Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Phase
Not applicable
Started
2024-10-28
Last updated
2026-08-12
Condition(s) studied
Wilson Disease
Investigational drug(s) / intervention(s)
MWAV201
MWAV201: Nonreplicating, recombinant gene transfer vector
Study summary
The primary objective of this study is to evaluate the tolerability and safety of MWAV201 in patients with Wilson disease.
Eligibility
Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria:
* Male or female aged 18 and 65 years inclusive;
* Confirmed diagnosis of Wilson disease;
* Low copper diet and standardized medication for ≥ 1 year;
* Stable Wilson disease for ≥ 1 year;
* Able to understand and willing to follow study procedures.
Exclusion Criteria:
* Significant hepatic inflammation as evidenced by liver function test.
* Liver biopsy or liver stiffness measurement show progressive liver fibrosis.
* Laboratory tests or clinical symptoms indicate decreased liver reserve function.
* Other chronic liver disease (such as hepatitis B).
* Any signs of decompensated liver function (such as ascites).
* History of liver transplant or plan to receive liver transplant.
* Other diseases with clinical significance, such as cardiovascular and cerebrovascular diseases, kidney diseases, respiratory system diseases, neurological diseases, mental illnesses, active infections, etc.
* Body Mass Index ≥ 30 kg/m2.
* Other conditions that, in the Investigator's opinion, may not be suitable for the subject to be enrolled.
Primary outcome measure(s)
Safety and tolerability profile [including adverse events (AEs) and serious adverse events (SAEs)] — At 1-Year post treatment AEs will be summarized based on incidence and the date of onset for the event.
Trial sites (1)
Facility
City
Region
Status
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai
Shanghai Municipality
More Xinhua Hospital, Shanghai Jiao Tong University School of Medicine trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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